Gene therapy is the amelioration of disease through the use of nucleic acid. This broad definition reflects the large spectrum of diseases that can be potentially considered for treatment, as well as the many methods of introducing the nucleic acid into target cells, which may or may not be directly affected by the disease. This chapter focuses on a very specific area of gene therapy research—the development and application of helper-dependent adenoviral vectors (HDAds) (also referred to as gutless, gutted, mini, fully deleted, high-capacity, A, pseudo) for gene therapy. Successful gene therapy requires a gene transfer vector that is able to efficiently transduce the target cells in vivo and provide high-level and long-term transgene expression in the absence of toxicity. How HDAds have measured up to these expectations is reviewed in this chapter.

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